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The trial is taking place at:
R

Rare Disease Research, LLC | Atlanta, GA

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Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)

P

PepGen

Status and phase

Enrolling
Phase 1

Conditions

Myotonic Dystrophy 1

Treatments

Other: Placebo
Drug: PGN-EDODM1

Study type

Interventional

Funder types

Industry

Identifiers

NCT06204809
PGN-EDODM1-101

Details and patient eligibility

About

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Enrollment

24 estimated patients

Sex

All

Ages

18 to 50 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
  • Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles
  • Presence of myotonia

Exclusion criteria

  • Congenital DM1
  • Known history or presence of any clinically significant conditions that may interfere with study safety assessments
  • Abnormal laboratory tests at screening
  • Medications specific for the treatment of myotonia within 2 weeks prior to screening
  • Percent predicted forced vital capacity (FVC) <40%

Note: Other inclusion and exclusion criteria may apply.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

24 participants in 4 patient groups, including a placebo group

Single Dose of PGN-EDODM1 at Dose Level 1
Experimental group
Treatment:
Drug: PGN-EDODM1
Single Dose of PGN-EDODM1 at Dose Level 2
Experimental group
Treatment:
Drug: PGN-EDODM1
Single Dose of PGN-EDODM1 at Dose Level 3
Experimental group
Treatment:
Drug: PGN-EDODM1
Single Dose of Placebo
Placebo Comparator group
Treatment:
Other: Placebo

Trial contacts and locations

8

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Central trial contact

PepGen

Data sourced from clinicaltrials.gov

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